Sarepta touts clean data for commercial grade Duchenne gene therapy, clearing key hurdle to PhIII

After a trial flop and an unexpected detour, Sarepta says its gene therapy for Duchenne muscular dystrophy looks ready to get back on the Phase III track.

A review of preliminary safety and expression data from 11 patients suggests that the material manufactured with their updated commercial process spurred consistent production of dystrophin, the biotech reported, while maintaining a clean side effect profile.